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EP 3 268 013 A1

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(43) Date of publication: 17.01.2018 Bulletin 2018/03 (21) Application number: 16710702.8 (22) Date of filing: 11.03.2016

(51) International Patent Classification (IPC):  A61K 35/17(2015.01) A61P 37/06(2006.01) A61P 35/00(2006.01) (86) International application number: PCT/EP2016/055353 (87) International publication number: WO 2016/146542 (22.09.2016 Gazette 2016/38)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: MA MD (30) Priority: 13.03.2015 EP 15159075 (71) Applicants: Fondazione Telethon 00185 Roma (IT) Ospedale San Raffaele S.r.l. 20132 Milano (IT) Fondazione Centro San Raffaele 20132 Milano (IT)

(72) Inventors: GREGORI, Silvia, Adriana 20132 Milano (MI) (IT) RONCAROLO, Maria, Grazia 20132 Milano (MI) (IT) (74) Representative: Trillat, Anne-Cecile et al De Simone & Partners S.p.A. Via Vincenzo Bellini, 20 00198 Roma 00198 Roma (IT)